Haemophilia B

Amarna Therapeutics announces appointment of Dr Henk Streefkerk as new CEO

Retrieved on: 
Wednesday, June 7, 2023
Streefkerk, Medicine, Heart failure, Clinical trial, CMO, Doctor of Philosophy, Haemophilia B, Actelion, Medical director, Novartis, MD, Patient, Haemophilia, Therapy, Henk, Pharmaceutical industry, Organon

LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.

Key Points: 
  • LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.
  • Dr Streefkerk has a distinguished career as CMO of several biotechs, including PIQUR Therapeutics, Cellprotera and Rivia, and brings over a decade of experience working in big pharma including Novartis, Actelion and Organon.
  • "We are very pleased to introduce Henk Streefkerk as our new CEO.
  • Henk will be instrumental in leading Amarna towards the first in human clinical trial," said Thomas Eldered, Chairman of Amarna's supervisory board.

Amarna Therapeutics announces appointment of Dr Henk Streefkerk as new CEO

Retrieved on: 
Wednesday, June 7, 2023
Streefkerk, Medicine, Heart failure, Clinical trial, CMO, Doctor of Philosophy, Haemophilia B, Actelion, Medical director, Novartis, MD, Patient, Haemophilia, Therapy, Henk, Pharmaceutical industry, Organon

LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.

Key Points: 
  • LEIDEN, Netherlands, June 7, 2023 /PRNewswire/ -- Amarna Therapeutics , a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, including Hemophilia B, today announces the appointment of Dr Henk Streefkerk as the Company's new Chief Executive Officer and Medical Director.
  • Dr Streefkerk has a distinguished career as CMO of several biotechs, including PIQUR Therapeutics, Cellprotera and Rivia, and brings over a decade of experience working in big pharma including Novartis, Actelion and Organon.
  • "We are very pleased to introduce Henk Streefkerk as our new CEO.
  • Henk will be instrumental in leading Amarna towards the first in human clinical trial," said Thomas Eldered, Chairman of Amarna's supervisory board.

XOMA Acquires Cashflow-Generating Asset for its Royalty and Milestone Portfolio

Retrieved on: 
Thursday, March 30, 2023
Growth, Haemophilia, Haemophilia B, Bank

EMERYVILLE, Calif., March 30, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), announced today it has acquired the commercial payment and a portion of the milestone rights to IXINITY® [coagulation factor IX (recombinant)], which is marketed by Medexus Pharmaceuticals for the control and prevention of bleeding episodes and postoperative management in people with Hemophilia B, from Aptevo Therapeutics.

Key Points: 
  • EMERYVILLE, Calif., March 30, 2023 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), announced today it has acquired the commercial payment and a portion of the milestone rights to IXINITY® [coagulation factor IX (recombinant)], which is marketed by Medexus Pharmaceuticals for the control and prevention of bleeding episodes and postoperative management in people with Hemophilia B, from Aptevo Therapeutics.
  • “Adding a new commercial payment stream will benefit XOMA’s shareholders as we build our base of consistent incoming cash flows to help accelerate our royalty aggregation strategy,” stated Brad Sitko, Chief Investment Officer at XOMA.
  • “IXINITY has an established niche in the Hemophilia B treatment paradigm, with near-term potential opportunities for growth, including label and geographic expansion.”
    Under the terms of the agreement, XOMA has acquired the full commercial payment stream and a portion of Aptevo’s milestone rights related to IXINITY for $9.6 million.
  • In the second quarter of 2023, XOMA will begin receiving a mid-single digit percentage payment stream on all IXINITY sales from January 1, 2023, into the first quarter of 2035 and also will be entitled to milestone payments.

uniQure announces the European Commission approval of the first gene therapy for adults with hemophilia B

Retrieved on: 
Tuesday, February 21, 2023
Joint, Science, Medication, European Commission, CSL, United Kingdom, CMA, IX, Medicine, Haemophilia, Factor IX, Civil service commission, Patient, Liver, European Economic Area, Food, Research, ASX, Risk, CSL Behring, Health care, Disease, Partnership, Bleeding, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Haemophilia B, Quality of life, ABR, Teaching hospital, Pharmaceutical industry, Medical device, Dietary supplement, EEA, EU, European

LEXINGTON, Mass. and AMSTERDAM, Feb. 21, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received conditional marketing authorization (CMA) from the European Commission for HEMGENIX® (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B.

Key Points: 
  • It is the first approved gene therapy for hemophilia B in the European Union (EU) and European Economic Area (EEA).
  • “The European approval of HEMGENIX represents another major milestone in the field of genomic medicine and innovation in the treatment of people living with hemophilia B,” said Matt Kapusta , chief executive officer of uniQure.
  • Treatments for moderate to severe hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules.
  • uniQure conducted the research and clinical development for the product, which included three clinical trials across 34 global sites and involving 67 adults with hemophilia B.

Global Hemophilia Treatment Market Report 2022 to 2030 - Featuring Ferring, Kedrion, Octapharma and Novo Nordis Among Others

Retrieved on: 
Thursday, February 2, 2023
World Blood Donor Day, Haemophilia, Coagulopathy, Government, Blood, Diagnosis, VIII, Plasma, Incidence, Research, Risk, Haemophilia B, Male, Haemophilia A, Pregnancy, Growth, Disease, XI, Bleeding, Prevalence, Infection, Factor XI, Pet industry, Vaccine, Pharmaceutical industry, Medical device, Marketing

The global hemophilia treatment market is segmented by hemophilia type into Hemophilia A, Hemophilia B and others.

Key Points: 
  • The global hemophilia treatment market is segmented by hemophilia type into Hemophilia A, Hemophilia B and others.
  • There are two main forms of hemophilia namely Hemophilia A and Hemophilia B. Hemophilia A is approximately four times more prevalent than hemophilia B.
  • The major growth drivers for hemophilia treatment market include rising diagnosis rate, increase focus on prophylactic treatment and development of novel treatment.
  • Hemophilia A is four times more prevalent than hemophilia B and thus dominates the global hemophilia treatment market.

Orsini Specialty Pharmacy Selected by CSL Behring as a Limited Distribution Partner for HEMGENIX®, the First and Only Gene Therapy for Hemophilia B

Retrieved on: 
Tuesday, December 20, 2022
URAC, Degenerative disease, Muscle, Patient, IX, NABP, ACHC, CSL, Haemophilia, CSL Behring, Disease, Joint Commission, Swelling, Pharmacy, Haemophilia B, L.L.C, Joint, Factor IX, Case management, Pain, Accreditation Commission for Health Care, Conditional sentence, Full, Male, ELK, Pharmaceutical industry, Orsini

Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births.

Key Points: 
  • Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births.
  • "We are deeply grateful to be selected by CSL Behring as a trusted partner to provide the first gene therapy for patients with hemophilia B," said Brandon Tom, Orsini's Chief Executive Officer.
  • Our experienced, compassionate Hemophilia Care Team is fully committed to working with payors and providers to ensure patients receive access to this life-changing therapy."
  • Providing patients with comprehensive and compassionate care since 1987, Orsini is a leading independent specialty pharmacy focused on rare diseases and gene therapies.

uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B

Retrieved on: 
Friday, December 16, 2022
Observation, European Medicines Agency, European Commission, CHMP, Bleeding, EMA, Swelling, Protein, CMA, Muscle, Diagnosis, Patient, Partnership, ASX, Factor IX, Food, Lance, FIX, Safety, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ABR, Pain, Committee for Medicinal Products for Human Use, Committee, Joint, Advanced Therapy Medicinal Product, AAV, Padua, Haemophilia, Royal commission, Haemophilia B, CSL, Medical device, Pharmaceutical industry

Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.1,2 uniQure conducted the multi-year clinical development program for etranacogene dezaparvovec and will leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization.

Key Points: 
  • Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.1,2 uniQure conducted the multi-year clinical development program for etranacogene dezaparvovec and will leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization.
  • “We are very pleased with this positive opinion recommending conditional marketing authorization for etranacogene dezaparvovec,” said Matt Kapusta , chief executive officer of uniQure.
  • “The program’s multi-year clinical journey began in Europe with our first-in-human Phase I/II study, and the favorable CHMP opinion moves etranacogene dezaparvovec one step closer to the European hemophilia B community.
  • Etranacogene dezaparvovec is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients.

Hemophilia Treatment Market Report 2022-2030: Growth Influencers Include Preference for Prophylactic Treatment, Rise in Diagnosis Rate, Significant Rise in Usage of Recombinant Coagulation Factor - ResearchAndMarkets.com

Retrieved on: 
Monday, December 12, 2022
Health, Biotechnology, Degenerative disease, World Blood Donor Day, Haemophilia, Pregnancy, Diagnosis, Plasma, Prevalence, Haemophilia A, Research, XI, Disease, Coagulopathy, Incidence, Haemophilia B, Government, Growth, VIII, Factor XI, Infection, Blood, Risk, Renewable energy, Vaccine, Agriculture, Marketing

The product types studied for analyzing the overall global hemophilia treatment market are majorly segmented into plasma derived coagulation factor concentrates, recombinant coagulation factor concentrates, antifibrinolytic agents and desmopressin.

Key Points: 
  • The product types studied for analyzing the overall global hemophilia treatment market are majorly segmented into plasma derived coagulation factor concentrates, recombinant coagulation factor concentrates, antifibrinolytic agents and desmopressin.
  • The global hemophilia treatment market is segmented by hemophilia type into Hemophilia A, Hemophilia B and others.
  • With increase in healthcare expenditure, preference for prophylactic treatment, rise in diagnosis rate, significant rise in usage of recombinant coagulation factor are major contributors in growth of haemophilia market.
  • Hemophilia A is four times more prevalent than hemophilia B and thus dominates the global hemophilia treatment market.

FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

Retrieved on: 
Wednesday, November 23, 2022
Factor IX, Cosmetics, Patient, Smokeless tobacco, Food, Disease, Public health, Blood, CSL, Haemophilia B, Quality of life, Joint, Dietary supplement, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Center for Biologics Evaluation and Research, Vaccine, Transaminase, Bleeding, Woman, Brain, Research, Use of Free and Open-Source Software (FOSS) in the U.S. Department of Defense, Human services, FDA, Prevalence, Haemophilia, IV, Office of Inspector General, U.S. Department of Health and Human Services, Liver, China Biologic Products, Multimedia, ABR, Priority review, Animal, Headache, Muscle, Safety, Pharmaceutical industry, Birth control

"Gene therapy for hemophilia has beenon the horizon for more than two decades.

Key Points: 
  • "Gene therapy for hemophilia has beenon the horizon for more than two decades.
  • "Today's approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia."
  • Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.
  • The prevalence of Hemophilia B in the population is about one in 40,000; Hemophilia B represents about 15% of patients with hemophilia.

U.S. Food and Drug Administration approves CSL's HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B

Retrieved on: 
Wednesday, November 23, 2022
Observation, IX, Lightheadedness, Swelling, Pediatrics, Factor IX, Joint, Nausea, Pain scale, Rash, Iron deficiency, Hepatocellular carcinoma, University, Research, Muscle, CSL Behring, Patient, Incidence, Blood, Caregiver, CSL, Death, Pain, Haemophilia B, Pyelonephritis, Vaccine, Pathology, Haemophilia, AAV, Bleeding, Headache, Semen, Shivering, Safety, Liver cancer, ASX, Disease, Risk, Research and development, Nephrology, Abdominal pain, Padua, ABR, Cell, Cardiogenic shock, Immunodeficiency, Professor, Food, Development, Tissue, Clinical trial, Flushing, Freedom, Dialysis, Biotechnology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Transplant, FDA, Lance, Liver, Coalition, Fatigue, R, Medical device, Pharmaceutical industry, Dietary supplement

KING OF PRUSSIA, Pa., Nov. 22, 2022 /PRNewswire/ -- Global biotechnology leader CSL (ASX: CSL) today announced that the U.S. Food and Drug Administration (FDA) approved HEMGENIX® (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.

Key Points: 
  • "We are thrilled to witness this milestone in hemophilia B treatment," shared Kim Phelan, Chief Operating Officer of The Coalition for Hemophilia B.
  • The FDA approval is supported by results from the ongoing HOPE-B trial, the largest gene therapy trial in hemophilia B to date.
  • CSL Behring, a CSL business, will make HEMGENIX available for eligible people with hemophilia B as soon as possible.
  • HEMGENIX is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.